Alexion Pharmaceuticals, Inc, announced that the Australian Government's Therapeutic Goods Administration has approved the use of Soliris (eculizumab) for the treatment of all patients in Australia with paroxysmal nocturnal hemoglobinuria (PNH), a rare, debilitating and life-threatening blood disorder defined by chronic red blood cell destruction, or haemolysis. Soliris is the first therapy approved in Australia for the treatment of PNH.
"Prior to this positive action by the government, there has not been an approved, safe and effective therapy for the treatment of patients with PNH in Australia," said professor Jeffrey Szer, director of Clinical Haematology, The Royal Melbourne Hospital. "Soliris reduced haemolysis in every patient treated in clinical studies. Regulatory approval is an important step towards providing Australian patients with this debilitating and life-threatening disease access to Soliris, the only approved treatment. With access to Soliris, physicians will be able to prevent the hemolysis that underlies the severe, progressive and life-threatening consequences of PNH."
"This marketing approval reflects Australia's recognition of the safety and efficacy of Soliris as a treatment for patients with PNH," said Leonard Bell, chief executive officer of Alexion. "Our Sydney-based organization will now work with the Australian Federal healthcare authorities to obtain reimbursement for Soliris so that Australian patients with PNH who can benefit from Soliris will have access to it." A decision regarding funding of Soliris therapy for eligible patients is expected by the end of 2009.
Soliris has been approved by the US Food and Drug Administration (March 2007), the European Commission (June 2007), Health Canada (January 2009) and Australia's Therapeutic Goods Administration (February 2009) as the first treatment for all patients with PNH, a rare, debilitating and life-threatening blood disorder defined by haemolysis, or the destruction of red blood cells.